Sorafenib Tosylate in Treating Patients With Progressive Metastatic Neuroendocrine Tumors

Phase 2

Completed

Conditions: Recurrent Gastrointestinal Carcinoid Tumor
Gastrinoma
Neuroendocrine Tumor
Pancreatic Polypeptide Tumor
Somatostatinoma
Glucagonoma
Metastatic Gastrointestinal Carcinoid Tumor
WDHA Syndrome
Insulinoma
Recurrent Islet Cell Carcinoma

Interventions: Drug: sorafenib tosylate

Registration Number: NCT00131911

Lead Sponsor: National Cancer Institute (NCI)

Brief Summary: This phase II trial is studying how well sorafenib tosylate works in treating patients with progressive metastatic neuroendocrine tumors. Sorafenib tosylate may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth and by blocking blood flow to the tumor.

Detailed Description: PRIMARY OBJECTIVES:

I. To determine the objective tumor response rate of BAY 43-9006 (sorafenib tosylate) in patients with advanced neuroendocrine tumors.

SECONDARY OBJECTIVES:

I. Adverse event rate(s). II. Progression free survival and time to progression. III. Improvement in circulating hormone levels. IV. Overall survival.

OUTLINE: This is a multicenter study. Patients are grouped into 2 separate analysis Groups according to tumor type (Group A: Carcinoid; Group B: Islet cell/other well-differentiated tumor). Each Group was independently evaluated for all study endpoints.

Patients receive oral sorafenib tosylate twice daily on days 1-28. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity.

After completion of study treatment, patients are followed every 3 months until disease progression and then every 6 months for up to 2 years from study entry.

Recruitment & Eligibility

Status: COMPLETED

Sex: All

Target Recruitment: 93

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

Study Type: INTERVENTIONAL

Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Group A (patients with carcinoid tumors)	sorafenib tosylate	Patients receive 400 mg oral sorafenib twice daily on days 1-28.
Group B (islet cell and other neuroendocrine tumors)	sorafenib tosylate	Patients receive 400 mg oral sorafenib twice daily on days 1-28.

Primary Outcome Measures

Name	Time	Method
Confirmed Response Rate	Duration of Treatment (Up to 2 years)	Confirmed response rate was defined using Response Evaluation Criteria In Solid Tumors (RECIST). A confirmed response is defined as a complete response (CR) or partial response (PR) observed on subsequent scans at least 4 weeks apart. Confirmed response rate was estimated by the number of successes divided by the total number of evaluable patients.\> \> Complete Response (CR) is defined as the disappearance of all target lesions.\> Partial Response (PR) is defined as a 30% decrease in sum of longest diameter of target lesions;\> \> We report the percentage of patients with a confirmed response and a 95% confidence interval estimated by the Duffy and Santner method.

Secondary Outcome Measures

Name	Time	Method
Progression Free Survival	Time from registration to progression or death (up to 2 years)	Progression was defined using Response Evaluation Criteria In Solid Tumors (RECIST) as a 20% increase in the su of longest diameter of target lesions. Progression free survival (PFS) was defined as the time from registration to progression or death of any cause. Participants who were progression free were censored at the date of their most recent disease assessment. The median PFS with 95% CI was estimated using the Kaplan Meier method.
Toxicity	Up to 2 years	For this secondary endpoint, toxicity is defined as a grade 3 or higher adverse events that is classified as either possibly, probably, or definitely related to study treatment. The assignment of attribution to study treatment and grade (or degree of severity) of the adverse event are classified using the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) version 3.0. The number of participants reporting a grade 3 or higher toxicity are reported.
Overall Survival	From registration to death (up to 2 years)	Overall survival (OS) was defined as the time from registration to death of any cause. Surviving patients were censored at the date of last follow-up. The median OS with 95% CI was estimated using the Kaplan Meier method.
Duration of Response	Time from response to progression (up to 2 years)	Duration of response (DOR) was defined as the time from attaining a response (PR or CR) to the date of progression. Participants without progression were censored at the date of their most recent disease assessment. The median DOR was estimated using simple summary statistics.